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JPWO2023026994A1 - - Google Patents

Info

Publication number
JPWO2023026994A1
JPWO2023026994A1 JP2023543884A JP2023543884A JPWO2023026994A1 JP WO2023026994 A1 JPWO2023026994 A1 JP WO2023026994A1 JP 2023543884 A JP2023543884 A JP 2023543884A JP 2023543884 A JP2023543884 A JP 2023543884A JP WO2023026994 A1 JPWO2023026994 A1 JP WO2023026994A1
Authority
JP
Japan
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
JP2023543884A
Other languages
Japanese (ja)
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed filed Critical
Publication of JPWO2023026994A1 publication Critical patent/JPWO2023026994A1/ja
Pending legal-status Critical Current

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/62Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being a protein, peptide or polyamino acid
    • A61K47/64Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • A61K38/1719Muscle proteins, e.g. myosin or actin
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/62Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being a protein, peptide or polyamino acid
    • A61K47/65Peptidic linkers, binders or spacers, e.g. peptidic enzyme-labile linkers
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0033Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being non-polymeric
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K7/00Peptides having 5 to 20 amino acids in a fully defined sequence; Derivatives thereof
    • C07K7/04Linear peptides containing only normal peptide links
    • C07K7/08Linear peptides containing only normal peptide links having 12 to 20 amino acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/314Phosphoramidates
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/323Chemical structure of the sugar modified ring structure
    • C12N2310/3233Morpholino-type ring
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • C12N2310/3513Protein; Peptide

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicinal Chemistry (AREA)
  • Organic Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Biomedical Technology (AREA)
  • Epidemiology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2023543884A 2021-08-21 2022-08-19 Pending JPWO2023026994A1 (el)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
JP2021135254 2021-08-21
PCT/JP2022/031434 WO2023026994A1 (ja) 2021-08-21 2022-08-19 ヒトトランスフェリンレセプター結合ペプチド-薬物コンジュゲート

Publications (1)

Publication Number Publication Date
JPWO2023026994A1 true JPWO2023026994A1 (el) 2023-03-02

Family

ID=85322137

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2023543884A Pending JPWO2023026994A1 (el) 2021-08-21 2022-08-19

Country Status (4)

Country Link
US (1) US20240390508A1 (el)
EP (1) EP4389893A1 (el)
JP (1) JPWO2023026994A1 (el)
WO (1) WO2023026994A1 (el)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12018087B2 (en) 2018-08-02 2024-06-25 Dyne Therapeutics, Inc. Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject
WO2020028864A1 (en) 2018-08-02 2020-02-06 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
WO2024195711A1 (ja) * 2023-03-17 2024-09-26 富士フイルム株式会社 環状ペプチド又はその塩及びそれらの利用

Family Cites Families (40)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US3687808A (en) 1969-08-14 1972-08-29 Univ Leland Stanford Junior Synthetic polynucleotides
US5264423A (en) 1987-03-25 1993-11-23 The United States Of America As Represented By The Department Of Health And Human Services Inhibitors for replication of retroviruses and for the expression of oncogene products
US5276019A (en) 1987-03-25 1994-01-04 The United States Of America As Represented By The Department Of Health And Human Services Inhibitors for replication of retroviruses and for the expression of oncogene products
EP0506830B1 (en) 1989-12-20 2005-12-14 Avi Biopharma, Inc. Uncharged morpholino-based polymers having phosphorous-containing chiral intersubunit linkages
US5525491A (en) 1991-02-27 1996-06-11 Creative Biomolecules, Inc. Serine-rich peptide linkers
US5955599A (en) 1995-06-01 1999-09-21 Hybridon, Inc. Process for making oligonucleotides containing o- and s- methylphosphotriester internucleoside linkages
HU228398B1 (en) 1999-02-12 2013-03-28 Daiichi Sankyo Company Novel nucleosides and oligonucleotide analogues
JP4148662B2 (ja) 2000-08-10 2008-09-10 第一三共株式会社 ヌクレオシド及びオリゴヌクレオチド類縁体を含有する核酸試薬及び医薬
JP2004528014A (ja) 2000-12-07 2004-09-16 イーライ・リリー・アンド・カンパニー Glp−1融合タンパク質
EP2530153B1 (en) 2002-11-25 2016-03-16 Masafumi Matsuo ENA nucleic acid drugs modifying splicing in mRNA precursor
DK2216407T3 (en) 2003-03-07 2016-03-29 Alnylam Pharmaceuticals Inc therapeutic compositions
ES2361325T3 (es) 2004-06-28 2011-06-16 The University Of Western Australia Oligonucleótidos antisentido para inducir la omisión de exón y métodos de uso de los mismos.
EP1964916B1 (en) 2005-12-06 2012-08-01 The University of Tokyo Multi-purpose acylation catalayst and use thereof
US20090326041A1 (en) 2006-05-05 2009-12-31 Isis Pharmaceuticals, Inc. Compounds and methods for modulating expression of sglt2
ES2603379T3 (es) 2006-10-09 2017-02-27 Roche Innovation Center Copenhagen A/S Compuestos antagonistas de ARN para la modulación de PCSK9
EP2410053B2 (en) 2006-10-18 2020-07-15 Ionis Pharmaceuticals, Inc. Antisense compounds
ES2479393T3 (es) 2007-11-15 2014-07-24 Sarepta Therapeutics, Inc. Método de síntesis de oligómeros de morfolino
WO2009147368A1 (en) 2008-06-04 2009-12-10 Medical Research Council Peptides
US8084601B2 (en) 2008-09-11 2011-12-27 Royal Holloway And Bedford New College Royal Holloway, University Of London Oligomers
WO2011052436A1 (ja) 2009-10-29 2011-05-05 国立大学法人大阪大学 架橋型人工ヌクレオシドおよびヌクレオチド
TWI541024B (zh) 2010-09-01 2016-07-11 日本新藥股份有限公司 反義核酸
AU2011367230B2 (en) 2011-05-05 2017-08-10 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
ES2876939T3 (es) 2011-08-30 2021-11-15 Res & Innovation Uk Péptidos penetradores de células que tienen un dominio hidrófobo central
CN104126010B (zh) 2011-12-16 2021-04-06 国立大学法人东京医科齿科大学 嵌合的双链核酸
CN110055243B (zh) 2011-12-28 2024-03-26 日本新药株式会社 反义核酸
ES2758451T3 (es) 2012-09-19 2020-05-05 Gemvax & Kael Co Ltd Péptido de penetración celular, conjugado que comprende el mismo y composición que comprende el conjugado
US10377789B2 (en) 2012-09-21 2019-08-13 Osaka University Oligonucleotide and artificial nucleoside having guanidine bridge
GB201314411D0 (en) 2013-08-12 2013-09-25 Medical Res Council Peptide conjugates
JP6233903B2 (ja) 2014-02-18 2017-11-22 国立大学法人大阪大学 架橋型ヌクレオシドおよびヌクレオチド
US20150329609A1 (en) * 2014-03-03 2015-11-19 Sujata Acharjee Chimeric dystrophin proteins to treat dystrophinopathies
WO2015168172A1 (en) 2014-04-28 2015-11-05 Isis Pharmaceuticals, Inc. Linkage modified oligomeric compounds
SG10201912858VA (en) 2014-06-17 2020-02-27 Nippon Shinyaku Co Ltd Antisense nucleic acids
US10723755B2 (en) 2014-11-18 2020-07-28 Zata Pharmaceuticals, Inc. Phosphoramidite synthones for the synthesis of self-neutralizing oligonucleotide compounds
EA039366B1 (ru) * 2015-06-24 2022-01-19 Джей Си Ар ФАРМАСЬЮТИКАЛЗ КО., ЛТД. Антитело к рецептору трансферрина человека, проникающее через гематоэнцефалический барьер
JP6823269B2 (ja) 2016-06-20 2021-02-03 株式会社GenAhead Bio 抗体−薬物コンジュゲート
CN111770757A (zh) * 2017-09-22 2020-10-13 艾维迪提生物科学公司 诱导外显子跳读的核酸-多肽组合物和方法
SG11202100934PA (en) 2018-08-02 2021-02-25 Dyne Therapeutics Inc Muscle targeting complexes and uses thereof for treating dystrophinopathies
CN113677374A (zh) 2019-04-08 2021-11-19 国立大学法人东京医科齿科大学 肌疾病治疗用药物组合物
WO2021054370A1 (ja) 2019-09-18 2021-03-25 国立大学法人東京医科歯科大学 核酸複合体
CN115151556A (zh) * 2020-02-22 2022-10-04 Jcr制药股份有限公司 人转铁蛋白受体结合肽

Also Published As

Publication number Publication date
WO2023026994A1 (ja) 2023-03-02
EP4389893A1 (en) 2024-06-26
US20240390508A1 (en) 2024-11-28

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